Rapid development of recently emerging precision medicine techniques represented by gene therapy has brought hope for the treatment of rare blinding eye diseases such as inherited retinal diseases (IRDs) for which there was no effective treatment previously. Although the globally growth of clinical trials for IRDs has increased rapidly over the past decade, due to the highly genetic and clinical phenotypic heterogeneity, as well as limited data on epidemiology and natural history of the disease, along with severe loss of vision function of majority patients for which the established measurements may not be appropriate, such studies lack standard outcome measurements and endpoints to asses clinical meaningful effectiveness, posing great challenges in terms of study design and evaluation of treatment efficacy, as well as clinical practice application. At present, there is no systematic nor standardized guidance on safety measures, clinical outcomes and endpoints of visual function for clinical trial design in IRDs. Therefore, in order to standardize the validated evaluation of IRDs clinical efficacy outcome measurements and endpoints, the Fundus Disease Group of Chinese Medical Association Ophthalmology Branch and Fundus Disease Committee of Chinese Medical Doctor Association Ophthalmology Branch organized domestic experts to put forward consensus and recommendations on standardizing outcome measurements and endpoints for clinical study design in IRDs, aiming to advance the study design of IRDs natural history research and clinical trials and to effectively evaluate disease progression and intervention efficacy. Along with the development of medical science and clinical trials, relevant content will be improved and updated accordingly.
Inherited retinal diseases (IRDs) are a group of severe retinal degenerative diseases leading to permanent visual impairment. IRDs are the major cause of irreversible blindness in children and working age groups. Gene therapy is a new clinical treatment method and currently the only clear and effective treatment for IRDs, while, there are still risks in clinical research and application. How to standardize perioperative management and reduce the potential risks of treatment is one of the keys to ensure the safety and effectiveness of treatment. However, there is no systematic and standardized guidance on the perioperative management for IRDs gene therapy. Therefore, in order to standardize the perioperative management, the Fundus Disease Group of Ophthalmology Society of Chinese Medical Association and Chinese Medical Doctor Association organized domestic experts to put forward standardized opinions on the perioperative management of IRDs gene therapy in China after repeated discussion and combined with domestic and foreign research experience, so as to provide clinicians with reference and application in clinical research and practice.
Based-on retrieval and reviewing information of evidence-based clinical practice guidelines from the literature, this paper introduced the key components of assessment guideline quality, including the process of development and reporting of guideline.
ObjectiveTo evaluate the quality of pancreatic cancer guidelines using evidence-based methods based on the global burden of pancreatic cancer, so as to explore its status, region distribution, characteristics of coverage themes, and difference of therapies recommended by the guidelines of various quality, and to provide references for clinical decisionmaking.
MethodsPubMed, The Cochrane Library (Issue 11, 2013), CBM, CNKI, and VIP, as well as the website of National Guidelines Clearinghouse (NGC), Guidelines International Network (GIN), and National Institute for Clinical Excellence (NICE) were systematically searched for pancreatic cancer treatment guidelines. The Appraisal of Guidelines for Research and Evaluation (AGREE Ⅲ) was applied to assess methodological quality of included guidelines.
ResultsA total of 14 relevant guidelines (including five evidence-based guidelines) were included involving seven countries of four continents (Asia, Europe, North America and Oceania) and four international academic organizations. There were only two domains, namely "scope and purpose" and "clarity of presentations" which got high average scores (more than 60%) among all 14 guidelines. The mean AGREE domain scores in guidelines varied with areas, and the quality of five evidence-based guidelines was superior to that established by consensus. According to the outcomes of AGREE Ⅲ, 11 guidelines were weakly recommended, while 3 were not recommended due to poor methodological quality. The subjects of 14 guidelines covered six treatment categories, including chemotherapy, surgery, radiotherapy, support therapy, radiotherapy, and interventional therapy.
ConclusionThe overall methodological quality of pancreatic cancer guidelines is not high among different countries or regions. The quality of evidence-based guidelines is superior to that established by consensus. Chemotherapy, surgery, radiotherapy and support therapy were reccommended as predominant choice by these guidelines.
Objective To systematically evaluate the efficacy and safety of iris-registration in wavefront-guided LASIK (IR+WG LASIK) versus conventional LASIK for correction of myopia accompanied with astigmatism. Methods Such databases as PubMed, EMbase, The Cochrane library (Issue 2, 2012), CBM, CNKI, VIP, and WangFang Data were searched to collect the randomized controlled trials (RCTs) and quasi-RCTs about IR+WG LASIK versus conventional LASIK for correction of myopia accompanied with astigmatism. The retrieval time was from inception to February 2012, and the language was in both Chinese and English. Two reviewers independently screened the literature, extracted the data and assessed the quality of the included studies. Then the meta-analysis was performed by using RevMan 5.1 software. Results A total of 9 studies involving 3 903 eyes were included. The results of meta-analysis showed that, compared with the conventional LASIK group, the IR+WG LASIK group had a higher ratio in patients with postoperative uncorrected visual acuity no less than 1.0 (RR=1.03, 95%CI 1.01 to 1.05, P=0.002), as well as in patients with best-corrected visual acuity gained over 1 line (RR=1.75, 95%CI 1.49 to 2.16, Plt;0.000 01); it was smaller in the postoperative high order aberration RMS (WMD=?0.16, 95%CI ?0.21 to ?0.11, Plt;0.000 01), coma-like RMS (WMD=?0.05, 95%CI ?0.11 to 0.00, P=0.07), spherical-like RMS (WMD=?0.15, 95%CI ?0.23 to ?0.07, P=0.000 2), and residual astigmatism (WMD=0.14, 95%CI 0.10 to 0.18, Plt;0.000 01); moreover, it was lower in the incidence of postoperative glare (RR=0.27, 95%CI 0.15 to 0.50, Plt;0.000 1), and it was higher in the subjective satisfaction of patients (RR=1.08, 95%CI 1.04 to 1.13, P=0.000 3). Conclusion Compared with conventional LASIK, IR+WG LASIK can more effectively reduce astigmatism, postoperative high order aberration RMS and spherical-like RMS. It can also get visual function including uncorrected visual acuity and best-corrected visual acuity, consequently increase patient’s satisfaction. But further studies are still required for its long-term effect.
In the process of guideline development and construction of clinical questions, it is necessary to guide clinicians to propose clinical problems into PICO (population, intervention, control, outcome) structured clinical questions. However, there are still unclear criteria to define and judge the appropriateness of the width of the PICO elements of a clinical question. Either too wide or too narrow can make the PICO question unsuitable to be a question for clinical practice guidelines to answer. We graded the clinical questions to be eight grades (3, 2, 1, 0, ?1, ?2, ?3, mixed) according to the number of the PIC elements, which obviously needed to be adjusted to evaluate applicability of the appropriateness of the width of the clinical questions. Our work can provide methodological references for clinicians and guideline developers.
The Universal Definition and Classification of Heart Failure consensus has proposed the universal definition, classification and staging criteria of heart failure. The prevalence of heart failure is still increasing, and the phenotype of heart failure with preserved ejection fraction (HFpEF) is becoming more and more common. Neuro-endocrine antagonists are effective in treating patients with heart failure with reduced ejection fraction (HFrEF). However, there is no effective drug that can improve the clinical prognosis of patients with HFpEF. The pathophysiological mechanism of HFpEF involves metabolic-inflammatory mechanism disorders, epicardial fat tissue accumulation, and coronary microvascular dysfunction. The exploratory treatment of these mechanisms requires further research to confirm whether it is beneficial to patients with HFpEF. In addition, the improvement of ejection fraction and the recovery of cardiac function in patients with HFrEF after treatment cannot interrupt the drug treatment of heart failure.
The number of clinical practice guidelines for traditional Chinese patent medicine has been increasing recently. However, the quality of guidelines was still low compared to international guidelines. Considering the characteristics of traditional Chinese patent medicine, we suggested the following items should be taken into account when developing traditional Chinese patent medicine guidelines: ensuring the standardized guidelines of traditional Chinese patent medicine research problem is scientific based on reliable evidence; identifying the common questions according to these research problems; understanding the strength of evidence and how to recommend correctly; inviting some experts in other fields to take part in the development of guidelines; paying more attention on the changes of disease burden and the impact of new methods and technologies when developing the guideline; paying more attention to the non-consensus opinions and evidence supporting these opinions; insisting on quality is the priority, while speed is secondary.
Recently, the National Cancer Center analyzed the disease burden and epidemiological trend of breast cancer based on the global population registration data, providing important reference for the prevention and control of breast cancer and health decision-making. Based on the current situation of prevention and control of breast cancer in China, this paper briefly interpreted the key points of the disease burden and trend of breast cancer.
